Risdiplam for treating spinal muscular atrophy

When NICE recommends a treatment as an option for use within a managed access agreement, NHS England will make it available according to the conditions in the managed access agreement. This means that, if a person has types 1, 2 or 3 spinal muscular atrophy (SMA) or pre-symptomatic SMA with 1 to 4 SMN2 copies, and the doctor responsible for their care thinks that risdiplam is the right treatment, it should be available for use, in line with NICE's recommendations and the criteria in the managed access agreement.

The Welsh ministers have issued directions to the NHS in Wales on implementing NICE technology appraisal guidance when the drug or treatment, or other technology, is approved for use within a managed access agreement. When a NICE technology appraisal recommends the use of a drug or treatment, or other technology, for use within a managed access agreement, the NHS in Wales must usually provide funding and resources for it within 2 months of the first publication of the final appraisal document or agreement of a managed access agreement by the NHS in Wales, whichever is the later.